For Patient

Patient Information

Oryzon has two drugs in clinical development, iadademstat (also known as ORY-1001), a selective LSD1 inhibitor for oncology, and vafidemstat (also known as ORY-2001), a dual LSD1-MAOB inhibitor for central nervous system (CNS) disorders.

Oryzon has completed one Phase I clinical trial with iadademstat (ORY-1001) in patients with acute leukemia, and especially in those with alteration in the MLL gene and other specific genomic alterations. Currently, Oryzon has two Phase IIa studies ongoing with iadademstat: the ALICE study, to evaluate the safety, tolerability, dose finding and efficacy of iadademstat in combination with standard treatment with azacitidine in newly diagnosed patients with Acute Myeloid Leukemia (AML) not eligible for intensive chemotherapy, and the CLEPSIDRA study, to evaluate the safety, tolerability, dose finding and efficacy of iadademstat in combination with platinum-etoposide in patients with small cell lung cancer.

With vafidemstat (ORY-2001), after completing one Phase I clinical trial in healthy volunteers, Oryzon is currently conducting and recruiting patients in three Phase IIa studies: the REIMAGINE study, on aggressiveness in patients with psychiatric disorders (Autism Spectrum Disorder, Borderline Personality Disorder and adult Attention Deficit Hyperactivity Syndrome) or neurodegenerative diseases (Alzheimer’s disease), the ETHERAL study in Alzheimer’s disease, and the SATEEN study in multiple sclerosis.

Both Oryzon’s experimental drugs target the Lysine-specific demethylase LSD1, a critical enzyme involved in the regulation of histone and chromatin remodeling. By doing this, they regulate important genes involved in the control of cellular differentiation, survival and proliferation.

Oryzon’s compounds are investigational medicines, meaning that they are being evaluated in clinical trials and are not yet available for use outside of a clinical trial setting. Approval by regulatory authorities such as the U.S. Food and Drug Administration (FDA) or the European Medicines Agency (EMA) at the end of their clinical development is the way to make our medicines available to the patients in need of these medicines.

In the meantime, our priority is to advance in the clinical trials in order to obtain the data required for the review and approval of our medicaments by the regulatory authorities. We provide below a summary of the clinical trials sponsored by Oryzon.

International and multicenter Phase IIa study to evaluate the safety, tolerability and preliminary efficacy of vafidemstat (ORY-2001) compared to placebo in patients with mild or moderate Alzheimer´s Disease.

The study is conducted in Spain, France and UK and the centers participating are:

“A pilot study to assess the safety, tolerability, dose finding and efficacy of ORY-1001 in combination with platinum-etoposide chemotherapy in patients with relapsed, extensive-stage disease small cell lung cancer”

Phase IIa study to evaluate the safety, tolerability, dose finding and efficacy of iadademstat (ORY-1001) in combination with platinum-etoposide chemotherapy in patients with relapsed, extensive-stage disease small cell lung cancer which are candidate to re-challenge with platinum-etoposide chemotherapy.

“A phase I study of Human Pharmacokinetics and Safety of ORY-1001, an LSD1 inhibitor, in relapsed or refractory acute leukemia”.

This is a study to establish the pharmacokinetics (how the human body processes the drug) and safety of the new anticancer drug ORY-1001 in cancer of blood and bone marrow after treatment or remission. This study included an extension arm on specific leukemia population for a preliminary efficacy evaluation. This clinical trial, which was conducted in 10 hospitals in Spain, France and United Kingdom, has finalized and preliminary results were presented at the American Society of Hematology (ASH) conference in December 2016. The publication of the final results in a scientific journal is in preparation.

"A Study to Assess the Safety, Tolerability and Pharmacokinetic of Single and Multiple Oral Doses of ORY-2001 in Healthy Male, Female Subjects and Elderly Population".

This Phase I study was performed at the Hospital de St. Pau in Barcelona in healthy volunteers to assess the safety and pharmacokinetics of the new drug ORY-2001. The study has been completed and the final results were presented at the Alzheimer Association International Conference (AAIC) in July 2017. The corresponding publication in a scientific journal is in preparation.